Publication: Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment (NSAA)

This research article, published in Muscle and Nerve, describes investigations into a well-developed analytical procedure for evaluating longitudinal treatment effects using the North Star Ambulatory Assessment (NSAA) data for illustration

• The analytical approach for evaluating the longitudinal treatment effects of ataluren vs. placebo and deflazacort vs. prednisone/prednisolone among placebo-treated patients in Duchenne muscular dystrophy (DMD) is described
• The comparison between the NSAA approach, which comprises 17 scorable items/outcomes to measure changes in motor function and a fixed-time analysis, is reviewed
• Potential benefits of evaluating disease burden by assessing the totality of treatment effects using multiple outcomes measures per patient, as per the NSAA, are addressed
• The feasibility of using this approach to shape future clinical trials in place of a fixed-time analysis is discussed

McDonald CM, Wei LJ, Flanigan KM, et al. Muscle Nerve. 2021;64(5):614–619

▼ Ataluren is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC at pharmacovigilance@ptcbio.com.

Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The European Summary of Product Characteristics for ataluren is available here. Please refer to your local country guidance for more information.

Deflazacort is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. PTC Therapeutics is the Marketing Authorization Holder for deflazacort in the US only. Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The US Prescribing Information for deflazacort is available here. Please refer to your local country guidance for more information.

For medical information, product complaints, or to report an adverse event, please call 1-866-562-4620 or email at usmedinfo@ptcbio.com. You may also report adverse events directly to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.